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Drug Shows Promise for Treatment of Stargardt Disease

Results from a study of fenretinide indicate that it may be a candidate for treatment of Stargardt disease.

The study was conducted by researchers from Sytera, Inc., a start-up biopharmaceutical company in La Jolla, California, and The Jules Stein Eye Institute, University of California at Los Angeles (UCLA). The study was funded, in part, by a grant from the Foundation.

Stargardt disease — the most common form of inherited macular degeneration — causes progressive vision loss in children and young adults, because of the accumulation of toxic cellular debris (lipofuscin) in the retinal pigment epithelium (RPE). The presence of lipofuscin prevents the RPE from performing its cellular support role, leading to degeneration of photoreceptors, which mediate vision.

Fenretinide — which was originally developed for the treatment of certain cancers, rheumatoid arthritis, acne, and psoriasis — inhibits the production and accumulation of a specific toxic vitamin A byproduct, A2E, within lipofuscin in the RPE. Inhibition of A2E formation should slow or arrest the accumulation of lipofuscin and the progression of visual loss in patients with Stargardt disease.

In the study conducted by Sytera and collaborators at The Jules Stein Eye Institute, a mouse model of Stargardt disease was used to test the efficacy of fenretinide. The investigators found that A2E accumulation was decreased by 60% in these mice after 28 days of treatment with fenretinide.

"The results are encouraging, and it's a nice payoff for the funding we provided along the research continuum for Stargardt disease," says Stephen Rose, chief research officer at the Foundation. "FFB-funded researchers discovered the ABCA4 gene, which causes the disease, and FFB-funded researchers developed the animal model for studies like Sytera's. Hopefully, it will pave the way for clinical trials in humans."